A Phase 3 Rollover Study of Lumacaftor in Combination With Ivacaftor in Subjects 12 Years and Older With Cystic Fibrosis

Recruiting in Palo Alto (17 mi)

+163 other locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Vertex Pharmaceuticals Incorporated

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Breakthrough Therapy

Trial Summary

What is the purpose of this trial?

The purpose of this study is to evaluate the efficacy and safety of long-term treatment with lumacaftor in combination with ivacaftor in people 12 years and older with Cystic Fibrosis.

Eligibility Criteria

Inclusion Criteria

Signed informed consent form (ICF), and where appropriate, signed assent form.

Participants entering the Part A Treatment Cohort: Completed 24 weeks of study drug treatment in Study 103 or Study 104 and elect to enroll in Part A treatment cohort.

Participants entering the Part B Treatment Cohort: Completed 56 days of study drug treatment in Cohort 4 of Study 102 and elect to enroll in Part B treatment cohort.

See 2 more

Treatment Details

Interventions

Ivacaftor (CFTR Modulator)
Lumacaftor Plus Ivacaftor Combination (CFTR Modulator)

Participant Groups

7Treatment groups

Experimental Treatment

Active Control

Group I: Arm 7 Part B: Placebo - LUM 400 mg q12h/ IVA 250 mg q12hExperimental Treatment1 Intervention

Participants who received placebo matched to LUM and IVA tablet in Cohort 4 of the previous study VX09-809-102, and will receive LUM 400 mg plus IVA 250 mg FDC tablet orally in the morning and evening in this study VX12-809-105 up to Week 96.

Group II: Arm 6 Part B: LUM 400 mg q12h/ IVA 250 mg q12hExperimental Treatment1 Intervention

Participants who received LUM 400 mg plus IVA 250 mg FDC tablet orally in the morning and evening in Cohort 4 of the previous study VX09-809-102, and will receive the same treatment in this study VX12-809-105 up to Week 96.

Group III: Arm 4 Part A: Placebo - LUM 400 mg q12h/ IVA 250 mg q12hExperimental Treatment1 Intervention

Participants who received placebo matched to LUM and IVA tablet in the previous study VX12-809-103 or VX12-809-104, and will receive LUM 400 mg plus IVA 250 mg FDC tablet orally in the morning and evening in this study VX12-809-105 up to Week 96.

Group IV: Arm 3 Part A: LUM 400 mg q12h/ IVA 250 mg q12hExperimental Treatment1 Intervention

Participants who received LUM 400 mg plus IVA 250 mg FDC tablet orally in the morning and evening in the previous study VX12-809-103 or VX12-809-104, and will receive the same treatment in this study VX12-809-105 up to Week 96.

Group V: Arm 2 Part A: Placebo - LUM 600 mg qd/ IVA 250 mg q12hExperimental Treatment2 Interventions

Participants who received placebo matched to LUM and IVA tablet in the previous study VX12-809-103 or VX12-809-104, and will receive LUM 600 mg plus IVA 250 mg FDC tablet orally in the morning and IVA 250 mg film-coated tablet orally in the evening in this study VX12-809-105 up to Week 96.

Group VI: Arm 1 Part A: LUM 600 mg qd/ IVA 250 mg q12hExperimental Treatment2 Interventions

Participants who received lumacaftor (LUM, VX-809) 600 milligram (mg) plus ivacaftor (IVA, VX-770) 250 mg fixed-dose combination (FDC) tablet orally in the morning and IVA 250 mg film-coated tablet orally in the evening, in the previous study VX12-809-103 or VX12-809-104, and will receive the same treatment in this study VX12-809-105 up to Week 96.

Group VII: Arm 5 Part A: Observational CohortActive Control1 Intervention

Participants who received either LUM 600 mg plus IVA 250 mg FDC tablet orally in the morning and IVA 250 mg film-coated tablet orally in the evening OR LUM 400 mg plus IVA 250 mg FDC tablet orally in the morning and evening OR placebo matched to LUM and IVA in the morning and evening, in the previous study VX12-809-103 or VX12-809-104, and will be observed (will not receive study drug) in this study VX12-809-105 for up to 2 years.

Ivacaftor is already approved in United States, European Union, Canada for the following indications:

🇺🇸 Approved in United States as Kalydeco for: